US approves new drug to manage sickle cell disease

AP – United States (US) regulators on Friday approved a new medicine that can help reduce extremely painful sickle cell disease flare-ups.

The Food and Drug Administration approved Novartis AG’s Adakveo for patients 16 and older.

The monthly infusion, which halves occurrences of sickle cell pain episodes, will be priced roughly USD85,000 to USD113,000 per year, depending on dosing. Insured patients generally will pay less.

Sickle cell disease is one of the most common inherited blood disorders, affecting about 100,000 Americans, most of them black, and about 300 million people worldwide.

Its hallmark is periodic episodes in which red blood cells stick together, blocking blood from reaching organs and small blood vessels. That causes intense pain and cumulative organ damage that shortens the lives of people with the disease.

“The duration and severity of these pain crises worsens with aging. Often patients die during one of these crises,” said Dr Biree Andemariam, chief medical officer of the Sickle Cell Disease Association of America.

Andemariam, a former Novartis advisory board member, said the drug appears to work better the longer patients receive it. The Swiss drugmaker is continuing patient testing to determine whether Adakveo, also known as crizanlizumab, lengthens patients’ lives, said Ameet Mallik, the company’s head of US oncology and blood disorders.

He said severe pain episodes send US patients to emergency departments about 200,000 times per year. About 85 per cent are hospitalised for days to a week, running up big bills.

The debilitating condition also causes anaemia, delayed growth, vision damage and painful swelling in hands and feet, making it hard for some people to maintain jobs or attend school.

Bottles of Novartis’s Adakveo. PHOTO: AP